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在生长激素激发试验前给予性类固醇作为启动治疗或在青春期生长延迟中作为促生长治疗的临床益处:欧洲内分泌罕见病研究网络(ENDO-ERN)中心的一项回顾性研究结果

Clinical benefits of sex steroids given as a priming prior to GH provocative test or as a growth-promoting therapy in peripubertal growth delays: Results of a retrospective study among ENDO-ERN centres.

作者信息

Galazzi Elena, Improda Nicola, Cerbone Manuela, Soranna Davide, Moro Mirella, Fatti Letizia Maria, Zambon Antonella, Bonomi Marco, Salerno Mariacarolina, Dattani Mehul, Persani Luca

机构信息

Department of Endocrine and Metabolic Diseases &, Lab of Endocrine and Metabolic Research, Istituto Auxologico Italiano IRCCS, Milan, Italy.

Department of Translational Medical Sciences, Pediatric Endocrinology Unit, Federico II University of Naples, Naples, Italy.

出版信息

Clin Endocrinol (Oxf). 2021 Feb;94(2):219-228. doi: 10.1111/cen.14337. Epub 2020 Oct 15.

Abstract

OBJECTIVES

Sex steroids, administered as a priming before GH stimulation tests (GHST) to differentiate between growth hormone deficiency (GHD) and constitutional delay of growth and puberty (CDGP) or as growth-promoting therapy using low-dose sex steroids (LDSS) in CDGP, are much debated. We aimed to compare auxological outcomes of CDGP or GHD children undergoing primed or unprimed GHST and to evaluate LDSS treatment in CDGP.

DESIGN

Retrospective study among three paediatric University Hospitals in Italy and UK.

METHODS

184 children (72 females) aged 12.4 ± 2.08 years underwent primed (/P ) or unprimed (/P ) GHST and were followed up until final height (FH). CDGP patients were untreated (CDG P ) or received LDSS (CDGP ). The cohort included 34 CDG P /P , 12 CDGP /P , 51 GHD/P , 29 CDG P /P , 2 CDGP /P and 56 GHD/P . FH standard deviation score (SDS), Δ SDS FH-target height (TH) and degree of success (-1 ≤ Δ SDS FH-SDS TH ≤ +1) were outcomes of interest.

RESULTS

GHD/P had better FH-SDS (-0.87 vs -1.49; P = .023) and ΔSDS FH-TH (-0.35 vs -0.77; P = .002) than CDGP /P . Overall, GHD/P showed the highest degree of success (90%, P = .006). Regardless of priming, both rhGH and LDSS improved degree of success compared to no treatment (89% and 86% vs 63%, P = .0009). GHD/P showed a trend towards a higher proportion of permanent GHD compared to GHD/P (30.43% vs 15.09%; P = .067).

CONCLUSION

In peripubertal children, priming before GHST improves diagnostic accuracy of GHST for idiopathic GHD. LDSS treatment improves auxological outcomes in CDGP.

摘要

目的

性类固醇在生长激素刺激试验(GHST)前作为激发剂用于区分生长激素缺乏症(GHD)和体质性生长和青春期延迟(CDGP),或在CDGP中作为低剂量性类固醇(LDSS)促生长治疗,一直存在很大争议。我们旨在比较接受激发或未激发GHST的CDGP或GHD儿童的体格学结果,并评估CDGP中的LDSS治疗。

设计

在意大利和英国的三家儿科大学医院进行的回顾性研究。

方法

184名年龄为12.4±2.08岁的儿童(72名女性)接受了激发(/P)或未激发(/P)的GHST,并随访至最终身高(FH)。CDGP患者未接受治疗(CDG P)或接受LDSS(CDGP)。该队列包括34名CDG P/P、12名CDGP/P、51名GHD/P、29名CDG P/P、2名CDGP/P和56名GHD/P。FH标准差评分(SDS)、ΔSDS FH-靶身高(TH)和成功程度(-1≤ΔSDS FH-SDS TH≤+1)是感兴趣的结果。

结果

GHD/P的FH-SDS(-0.87对-1.49;P = 0.023)和ΔSDS FH-TH(-0.35对-0.77;P = 0.002)优于CDGP/P。总体而言,GHD/P显示出最高的成功程度(90%,P = 0.006)。无论是否激发,与未治疗相比,重组人生长激素(rhGH)和LDSS均提高了成功程度(89%和86%对63%,P = 0.0009)。与GHD/P相比,GHD/P显示出永久性GHD比例更高的趋势(30.43%对15.09%;P = 0.067)。

结论

在青春期前儿童中,GHST前激发可提高GHST对特发性GHD的诊断准确性。LDSS治疗可改善CDGP的体格学结果。

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